Study of Pomalidomide in Subjects with MPN-Associated Myelofibrosis

• Research type

  Research Study

• Full title

  A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study To Compare Efficacy and Safety of Pomalidomide in subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence

• IRAS ID

  59944

• Contact name

  MF McMullin

• Sponsor organisation

  Celgene Corporation

• Eudract number

  2010-018965-42

• Research summary

  A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence.Blood cell production breaks down in patients who suffer with Myeloproliferative Neoplasm (MPN), a disorder closely linked to leukaemia where there is excess production of one or more type of blood cell in the bone marrow. There are three main types of MPN and this study is looking at one of them: myelofibrosis, where the bone marrow is initially overactive but then develops scar tissue and not enough blood cells are produced.A common side effect of patients with this disorder is anaemia. These patients will require regular red blood cell (RBC) transfusions. The purpose of this study is to compare the effectiveness of an experimental drug, pomalidomide, against placebo (a sugar pill) in aiding patients with the disorder to become independent of RBC-transfusions.This study will be conducted in sites in North America, Europe and Australia and approximately 210 people will take part. Eligible participants will be randomly assigned onto one of the two treatment arms: pomalidomide (approx 67%) or placebo (approx 33%).In the UK, the study will be conducted by doctors in the Haematology Departments at NHS hospitals.Participants will be on double-blind treatment (doctor or participant don??t know which treatment group the participant has been assigned to) for up to 6 months. After that period, only those participants who were randomised onto pomalidomide may continue to take it open-label (doctor and participant know that they are receiving pomalidomide) until they require a red blood cell transfusion. Participants will continue to be contacted by telephone to discuss their illness and current medications for a minimum of 5 years following randomisation. The total length of study participation could be up to 6.5 years.

• REC name

  HSC REC B

• REC reference

  10/NIR02/45

• Date of REC Opinion

  3 Nov 2010

• REC opinion

  Further Information Favourable Opinion